1. Market Insights, Epidemiology and Market Forecast-2028 SICKLE CELL DISEASE

2. Sickle Cell Disease, also known as sickle cell anemia, includes group of disorders affecting the red blood cells in the body. Sickle cell disease is irreversible that affects males and females equally, and is responsible for increased morbidity and mortality in affected persons. It is an inherited blood disorder characterized by defective hemoglobin. In this disease, there aren't enough healthy red blood cells to carry adequate oxygen throughout the body. 2 Sickle Cell Disease DelveInsight © 2019 | All rights reserved

3. There were approximately 133,385 Sickle Cell Disease prevalent cases in 2017 in the 6MM. The United States accounts for the highest Sickle Cell Disease prevalent population followed by the EU5 countries. Among the EU5 countries, France has the highest prevalent population with 15,000 cases followed by the United Kingdom, which has a prevalent population of 13,000 in 2017. There is no Sickle Cell Disease prevalence in Japan since the disease doesn’t occur in the Japanese population 3 Sickle Cell Disease Epidemiology DelveInsight © 2019 | All rights reserved

4. 4 Sickle Cell Disease Market Sickle Cell Disease market size was majorly dependent on the use of Hydroxyurea till 2017, when Endari was launched. The approval of the Emmaus’s Endari for treating Sickle Cell Disease complications in the United States in 2017 had marked a significant advancement in Sickle Cell Disease treatment. Companies are working on novel mechanisms of action (Pan-Selection Inhibitor) will further fuel the Sickle Cell Disease market growth. Most of Sickle Cell Disease treatments are dominated by off label and off patent medications (HU; Iron Chelators, Analgesics, Antibiotics, vaccines), to address the complications or for specific Sickle Cell Disease symptoms. Hydroxyurea was the first ever drug approved for Sickle Cell Disease treatment in 1998. The most distressing issue with Hydroxyurea was the lack of FDA approval for infants with Sickle Cell Disease in the United States.

5. Company- GBT Therapeutics Drug- Voxelotor/ GBT-440 01020304 5 DelveInsight © 2019 | All rights reserved Sickle Cell Disease Emerging Drugs and Companies Company- Pfizer Drug- Rivipansel Company- Novartis Drug- Crizanlizumab Company- Imara Therapeutics Drug- IMR-687

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